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Shionogi starts phase 2 clinical trial for Pompe Disease

The rare disease affects around one in every 22,000 people worldwide

Pharma website governance

Why pharma website projects stall long before compliance becomes the problem

Pharma teams often blame compliance when digital projects slow down. In reality, the bigger issue is usually weaker ownership, unclear review models and content structures that were never designed for...

Genetic Digital

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PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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Smarter Learning and Better Outcomes: How AI Can Provide an Advantage in Medical Education

See how AI-powered patient avatars allow physicians to test complex diagnoses and treatment plans in a simulated environment.

Impetus Digital

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UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

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Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

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New Webinar: 2026 Engagement Strategies For Modern Pharma

Turn interaction into intelligence! Don’t miss this chance to lead the industry with our free pharma-exclusive webinar!

Impetus Digital

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Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

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FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

Healthcare setting

Clinical research in 2026: The trends shaping tomorrow

Discover the key trends shaping clinical research in 2026, from patient-centric approaches to empowering site staff and fostering stronger human connections.

Cuttsy + Cuttsy

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AstraZeneca announces $2bn investment in US manufacturing

The new and expanded facilities will support production of rare disease drug treatments

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AstraZeneca reports positive results from rare disease trial

HES is a group of rare disorders characterised by persistently elevated levels of white blood cells

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